First gene therapy in Europe

02.11.2012 - Amsterdam-based uniQure biotherapies B.V. has received the first market authorisation for a gene therapy in the western world.

Today, the European Commission confirmed the EMA’s recommendation for market authorisation for the gene therapy Glybera (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD) suffering from recurring acute pancreatitis. The rare, inherited disease affects about 350-700 patients in Europe. Patients are unable to metabolise fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), a potentially lethal condition. Up to now, no gene therapy has been approved in the EU or the US. Glybera consists of an Adeno-associated Virus (AAV) vector expressing the faulty LPL enzyme.

“This therapy will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume”, commented Professor John Kastelein from Academic Medical Center of the University of Amsterdam. “By helping to normalise the metabolism of fat, Glybera prevents inflammation of the pancreas, thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities.”

Uniqure’s CEO Jörn Aldag stressed that Glybera is a proof-of-concept for the company’s gene therapy platform: “The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need.” Experts estimate the uniQure’s virus-based gene substitution therapy Glybera could spend €1m annually per patient. “Prices for orphan drugs like Provenge, Cerezyme or Naglazyme range from a 100,000-450,000 annually,” Aldag told EuroBiotechNews. “Since our therapy has already proven efficacy over several years after just one treatment, I think it's reasonable to start at the high end of this range.

In a next step, uniQure will go to special hospitals in Europe where patients with acute pancreatitis are treated and provide the doctors with its companion diagnostics chip. The firm, that took over the IP from Amsterdam Molecular Therapeutics (AMT), aims at identifying patients in about 20 hospitals throughout the EU.

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